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Net‐benefit regression with censored cost‐effectiveness data from randomized or observational studies

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Cost‐effectiveness analysis is an essential part of the evaluation of new medical interventions. While in many studies both costs and effectiveness (eg, survival time) are censored, standard survival analysis techniques… Click to show full abstract

Cost‐effectiveness analysis is an essential part of the evaluation of new medical interventions. While in many studies both costs and effectiveness (eg, survival time) are censored, standard survival analysis techniques are often invalid due to the induced dependent censoring problem. We propose methods for censored cost‐effectiveness data using the net‐benefit regression framework, which allow covariate‐adjustment and subgroup identification when comparing two intervention groups. The methods provide a straightforward way to construct cost‐effectiveness acceptability curves with censored data. We also propose a more efficient doubly robust estimator of average causal incremental net benefit, which increases the likelihood that the results will represent a valid inference in observational studies. Lastly, we conduct extensive numerical studies to examine the finite‐sample performance of the proposed methods, and illustrate the proposed methods with a real data example using both survival time and quality‐adjusted survival time as the measures of effectiveness.

Keywords: censored cost; cost; cost effectiveness; net benefit

Journal Title: Statistics in Medicine
Year Published: 2022

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